Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 5
- 主要终点
- decrease of number of cutaneous bullosa
研究概览
简要总结
Dystrophic epidermolysis bullosa hereditaria are genodermatosis responsible for formation of cutaneous bullous lesion arising spontaneously or after mechanical trauma.
These lesions are due to mutation on gene COL7A1 coding for collagen VII. There is no treatment available. Cares are consisting to dress lesions and to protect the skin.
The investigators have recently observed on patients having residual expression of collagen VII that phenotype severity is modulated by activation degree of dermic metalloproteinase. The investigators have also observed that epigallocatechin-3-gallate (Polyphenon E®) could be regulated this activity.
The primary purpose of this study is to assessing the efficacity of Polyphenon E to decrease the number of cutaneous bullosa after four month of treatment.
The primary outcome measure is the rate of patient presenting a decrease of 20% or more of the number of cutaneous bullosa.
Secondary outcomes are: severity of mucosa impairment, affected cutaneous surface, the average duration of cicatrisation and treatment tolerance.
This study foresees the inclusion of 22 patients older than 2 years old in 5 centers.
When patients are included, they will be randomized and receive the treatment (or placebo) for 4 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 2 Years 至 —(Child, Adult, Older Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •known mutation of COL7A1
排除标准
- •tea drinkers
- •patient receiving induction treatment,protease inhibitor treatment
- •liver failure
研究组 & 干预措施
1
patients receive the treatment in first and placebo in second part of study
干预措施: Polyphenon E before Placebo (Drug)
2
patients receive placebo in first and treatment in second part of study
干预措施: placebo before treatment (Drug)
结局指标
主要结局
decrease of number of cutaneous bullosa
时间窗: after 4 months of treatment
次要结局
- efficacity of treatment(at 4 , 6, 7 10 months after beginnig of treatment and at year 1)
- tolerance tio treatment(at 1, 4, 6, 7, 10 and 12 months after beginnig the treatment)
